Kanuma (sebelipase alfa) is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme.
Kanuma is specifically indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
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Adverse effects associated with the use of Kanuma may include, but are not limited to, the following:
Patients with Rapidly Progressive Disease Presenting within the First 6 Months of Life (≥30%):
Pediatric and Adult Patients (≥8%):
The recommended dose is as follows: Patients with Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life: The recommended starting dosage is 1 mg/kg administered once weekly as an intravenous infusion. For patients who do not achieve an optimal clinical response, increase to 3 mg/kg once weekly. Pediatric and Adult Patients with LAL Deficiency: The recommended dosage is 1 mg/kg administered once every other week as an intravenous infusion.